dm+d

Unassigned

New Medicines

ASCENIV (US)Primary immunodeficiency

Information

ASCENIV (US)
New molecular entity
Not known
ADMA Biologics

Development and Regulatory status

None
None
Marketed
Jan 21No indication that company plan to launch outside US. [6,7]
Oct 19Launched in US for immunodeficiency [5].
Sep 18After receiving updated improved compliance status from the FDA of its manufacturing facility, ADMA Biologics announce intention to resubmit BLA application for RI-002 in the US in Q3 2018 [4].
May 18ADMA Biologics announces intention to resubmit BLA to US FDA for Immunodeficiency disorders in 2018 [3]
Jul 16US FDA issues a Complete Response Letter (CRL). The CRL did not cite any concerns with the clinical safety and efficacy data for RI-002 submitted by ADMA in the BLA, nor has the FDA requested any additional clinical studies be conducted prior to FDA approval of the drug. The FDA identified in the CRL certain outstanding inspection issues and deficiencies at ADMA third-party contract manufacturers, including its contract drug substance and product manufacturer, its contract fill and finisher and compliance issues with a third-party contract testing laboratory, and requested documentation of corrections for a number of those issues. ADMA will work with its third party drug substance and product manufacturer, its contract fill and finish provider and a third-party contract testing laboratory to monitor their efforts in addressing and resolving outstanding issues relating to the observations. ADMA Biologics will work with the agency to reach an agreement on acceptable language for the package insert and container and vial labeling, if and when approval is granted [4].
Jul 15Biologics License Application submitted to the FDA for marketing authorization of RI-002 for primary immunodeficiency. The FDA has a 60-day review period to determine whether the BLA submission is complete and acceptable for filing. [2]
Dec 14ADMA Biologics announced positive preliminary data for PIII trial of RI-002 pts with Primary immunodeficiency disease (PIID). ADMA is currently assembling its submission for the FDA for Q1 or 2 of 2015 [1].

Category

Immunoglobulins
Human derived polyclonal antibodies which support the immune system to neutralize microbes [1].
1 December 2019The overall incidence of symptomatic primary immunodeficiency is estimated to be 1 per 10,000.
Primary immunodeficiency
Intravenous

Trial or other data

Dec 14The PIII US based clinical study (ADMA-003) enrolled 59 pts with PIDD who received RI-002 for 12 months. Preliminary analysis indicated that using RI-002 resulted in no serious bacterial infections (SBI) which is well under the FDA requirement of =1 SBI per pt-year and therefore the trial successfully achieved its primary endpoint. RI-002 was well tolerated with no reported serious adverse events attributable to RI-002. Final data from the study, including secondary endpoints such as IgG levels, lost days of work and number of visits to hospital, will be reported during the first quarter of 2015 [1].